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NIH grant 1R01FD008219-01A1: A retrospective and prospective study of leiomyosarcoma systemic therapy outcomes informed by tumor genomics
Institute: {'code': 'FD', 'abbreviation': 'FDA', 'name': 'Food and Drug Administration', 'admin_org_id': '12500799', 'admin_funding_url': ''}. PI: CRISTINA R ANTONESCU. Award: $345343.
Start: 2026-09-25T00:00:00.
ABSTRACT
The treatment of cancer is changing with the increasingly detailed understanding of cancer
genomics. For patients with sarcoma, our understanding lags behind more common cancers,
both from the rarity (< 1 % of all cancers in humans) and heterogeneity of sarcomas. This
heterogeneity is manifested by 70 or so histologic subtypes, many with translocations, for which
there are over 400 recognized. We seek to understand one of the most common forms of
sarcoma better, specifically leiomyosarcoma (LMS). Between 2500-3000 people will get
leiomyosarcoma arising in different parts of the body this year (uterus, branches of major veins,
soft tissue as three common primary sites). Patients with leiomyosarcoma also represent the top
accruing diagnosis for metastatic sarcomas in clinical trials due to frequent occurrence of lung
and other metastases, which makes leiomyosarcoma the most common fatal form of sarcoma.
While we have a general understanding of which drugs are active in this diagnosis, we do not
understand how the molecular genetics of leiomyosarcoma affect response or resistance to
systemic therapy. We will link what we know from our next-generation cancer sequencing
results to the systemic therapies we have given to patients for metastatic disease at our cancer
centers. We presently have access to over 400 patients’ sequencing data, and we expect to
sequence approximately 150 new patients a year between two sites. We will compare this
information to the systemic therapy those patients have received, as we develop a prospective
database starting with the patients we have already treated. In doing this, we will develop tools
to aid in risk stratification that we will make available publicly. The ultimate goal of our work is a
clinical trial to compare physician choice vs tool-selected treatment. This work will lay the
foundation for such a clinical trial.document fetched 2026-10-04 02:43